NCT03559062

Brief Summary

This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
67

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started May 2018

Shorter than P25 for phase_3

Geographic Reach
9 countries

27 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 17, 2018

Completed
19 days until next milestone

First Submitted

Initial submission to the registry

June 5, 2018

Completed
10 days until next milestone

First Posted

Study publicly available on registry

June 15, 2018

Completed
6 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 21, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 21, 2018

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

February 11, 2020

Completed
Last Updated

February 11, 2020

Status Verified

February 1, 2020

Enrollment Period

7 months

First QC Date

June 5, 2018

Results QC Date

December 20, 2019

Last Update Submit

February 4, 2020

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

  • Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8

    LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.

    From baseline through Week 8

Secondary Outcomes (3)

  • Absolute Change in Sweat Chloride At Week 8

    From baseline at Week 8

  • Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8

    From baseline through Week 8

  • Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit

    From first dose of study drug up to safety follow-up visit (up to Week 12)

Study Arms (3)

Placebo

OTHER

Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks.

Drug: Placebo

TEZ/IVA

EXPERIMENTAL

Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.

Drug: TEZ/IVADrug: IVADrug: Placebo

Ivacaftor

EXPERIMENTAL

Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.

Drug: IVADrug: Placebo

Interventions

TEZ/IVADRUG

Participants weighing \<40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing ≥40 kg received TEZ 100 mg/IVA 150 mg FDC tablet.

Also known as: VX-661/VX-770, tezacaftor/ivacaftor fixed dose combination
TEZ/IVA
IVADRUG

Participants weighing \<40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet.

Also known as: VX-770, ivacaftor
IvacaftorTEZ/IVA
PlaceboDRUG

Placebo matched to TEZ/IVA FDC

IvacaftorPlacebo

Eligibility Criteria

Age6 Years - 11 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
  • Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
  • Participants with a screening LCI2.5 result ≥7.5.
  • Participants who are able to swallow tablets.

You may not qualify if:

  • Clinically significant cirrhosis with or without portal hypertension.
  • Colonization with organisms associated with a more rapid decline in pulmonary status.
  • Solid organ or hematological transplantation.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (27)

Hunter Medical Research Institute (HMRI)

New Lambton Heights, Australia

Location

Princess Margaret Hospital for Children

Perth, Australia

Location

Lady Cilento Children's Hospital

South Brisbane, Australia

Location

The Children's Hospital at Westmead

Westmead, Australia

Location

Universitair Ziekenhuis Brussel - Campus Jette

Brussels, Belgium

Location

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

Leuven, Belgium

Location

University of Copenhagen Rigshospitalet

Copenhagen, Denmark

Location

Groupe Hospitalier Pellegrin - Hôpital des Enfants

Bordeaux, France

Location

Hôpital Necker - Enfants Malades

Paris, France

Location

Universitaetsklinikum Koeln

Cologne, Germany

Location

Universitaetsklinikum Essen

Essen, Germany

Location

Klinikum der Johann Wolfgang Goethe-Universitaet

Frankfurt, Germany

Location

Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen

Giessen, Germany

Location

Medizinische Hochschule Hannover

Hanover, Germany

Location

Universitaetsklinikum Heidelberg

Heidelberg, Germany

Location

Universitaetsklinikum Jena

Jena, Germany

Location

Universitaetsklinikum Tuebingen

Tübingen, Germany

Location

Our Lady's Children's Hospital

Dublin, Ireland

Location

University Hospital Limerick

Limerick, Ireland

Location

Klinika Mukowiscydozy, Oddział Chorób Płuc SZP ZOZ

Dziekanów Leśny, Poland

Location

Inselspital - Universitaetsspital Bern

Bern, Switzerland

Location

Kinderspital Zuerich

Zurich, Switzerland

Location

Royal Hospital for Sick Children

Edinburgh, United Kingdom

Location

Leeds General Infirmary

Leeds, United Kingdom

Location

Royal Brompton Hospital

London, United Kingdom

Location

Nottingham University Hospital City Campus

Nottingham, United Kingdom

Location

Southampton General Hospital

Southampton, United Kingdom

Location

Related Publications (2)

  • Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.

    PMID: 37983082DERIVED

  • Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.

    PMID: 33331662DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

tezacaftorivacaftor

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Results Point of Contact

Title
Medical Monitor
Organization
Vertex Pharmaceuticals Incorporated
medicalinfo@vrtx.com
617-341-6777

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 5, 2018

First Posted

June 15, 2018

Study Start

May 17, 2018

Primary Completion

December 21, 2018

Study Completion

December 21, 2018

Last Updated

February 11, 2020

Results First Posted

February 11, 2020

Record last verified: 2020-02

Locations

GB(5)FR(2)DK(1)CH(2)PL(1)BE(2)IE(2)AU(4)DE(8)